The journey for medicines seeking full regulatory approval is a long one – a timeline that is most keenly felt by the very patients waiting at the end of the pipeline, often without many, if any, viable alternatives. For many of these patients, daily quality of life is severely impacted by the intense challenges brought about by their disease.
In the case of chronic, debilitating, and even life-threatening diseases, ensuring patients can access novel and emerging treatments earlier in the approvals process in an otherwise sparse landscape can make an incredible difference to their all-round wellbeing. Herein lies the foundation of pre-launch programmes such as the Early Access to Medicines Scheme (EAMS), which supports those patients with direct access to potentially life-changing medicines and benefits the broader patient population through accelerated and more in-depth safety and efficacy evidence generation to support approvals.
Patients and pharmaceutical organisations alike may in turn see boosts to wider awareness across public, clinical, and research communities, driving development and strengthening future uptake of treatments that deliver true benefit to patients upon receiving regulatory approval for commercial launch.
‘Our work with leading life science partners, particularly in rare conditions and those that have seen little therapeutic innovation for decades, has highlighted the growing importance of programmes such as EAMS in ensuring that the treatments that really work reach the patients that need them most. Time and time again, we’ve seen just how critical powerful real-world evidence – beyond traditional follow-up periods and endpoints – are to driving regulatory approvals.’
The impact of these therapies on a quality of life, physiological, and real-time level are crucial to monitoring and demonstrating the prospective benefits and risks to patients, thus creating a fuller picture of a treatment’s potential.
How can technology help?
Advancements in technology over recent years have greatly improved the quality of data collected for clinical trials, with significant potential across a wide range of domains. Already, RwHealth’s work at Eleven Health in real-time monitoring for patients with Sickle Cell Disease (SCD) has unlocked the ability to change patient lives through daily disease management and predictive insights, at their fingertips. This is supported by FDA cleared and CE marked wearable devices that allow for automated and remote monitoring of key biometrics linked to health and wellbeing – sleep quality, activity, heart rate, blood oxygen saturation, and ECGs.
This approach has seen incredible improvements to patient engagement and retention, the resulting live data creating new sources of day-to-day information and more granular endpoints, without additional burden to patients. This real-world evidence base holds the key to demonstrating therapeutic efficacy and safety at a level not typically seen and is merged alongside base medical records, depth of diagnosis episodic data, as well as patient-reported outcomes such as EQ-5D and daily pain scores – derived from their dedicated patient portal.
Supporting the fight against COVID
RwHealth’s work in SCD has seen a great deal of focus around vaccination in what is an extremely high-risk patient group with severe forms of infection and mortality rates much more prevalent in those with the disease. However, public uncertainty around vaccine safety is an issue that has by no means bypassed even the most high risk of patients; many with specific conditions have voiced concerns around the vaccine’s safety and impact.
As such, RwHealth’s recent work has tracked this impact in real-time, applying both their patient-reported outcomes portal and remote monitoring wearable technology to track the real-time impacts of COVID and influenza vaccinations. Following daily patient journeys in terms of pain, psychological wellbeing, symptoms, as well as automated sleep and activity biometrics, RwHealth’s advanced analytics platform has captured real-world evidence for each vaccination’s effect, and any variation from patient baselines.
Indeed, this work has highlighted that any effects seen remain well in line with those of traditional vaccinations, such as flu, and may even have a lesser impact across the second and booster COVID vaccinations.
‘Our goal has been to help patients make informed decisions around their health, supporting the improvement of patient confidence and vaccine uptake in light of the ongoing risks of the pandemic through real-world evidence.’
As new therapeutics and vaccinations continue to emerge, it is this very ethos of demonstrable safety and efficacy that RwHealth works to apply to strategic partnerships centred around EAMS programmes.
‘Data science and AI solutions offer unique opportunities for monitoring patient outcomes, mapping this against specific treatments for better understanding of their real-world impact. I truly believe that Eleven’s ability to monitor live patient biometrics, alongside the integration of a real-time patient-reported outcomes portal, will be vital in supporting a deeper depth of data collection and analytic insight as part of EAMS.’ – Consultant Psychologist, Confidential NHS Trust
Mapping patient quality of life against key events and metrics
Patient-reported outcomes are a crucial part of understanding patient experiences and quality of life as they embark on their treatment journeys. Through the Eleven Health platform, this data can be easily input by patients as frequently as needed, far beyond what is currently recorded in limited clinic visits. With more frequent data points there comes the ability to map these against specific measurements and events — building a full picture of the impact of sleep upon quality of life.
As such, this provides the ability to apply live, remote monitoring methods to track and identify correlations between both automated validated, patient-reported, mood-based measures, such as the EQ-5D, and treatments or clinical events.
The creation of a daily timeline of patient-reported outcomes both across the cohort and at a patient level allows us to pinpoint the impact of key events upon the associated quality of life experienced by patients.
‘Our research and deep platform insights, married with discussions with patients and clinicians, have additionally flagged sleep as an important player in monitoring wellbeing and predicting the potential onset of complications’
Eleven’s work to date has highlighted key correlations between sleep and pain, supported by past publications that highlight the prevalence of sleep disorders in patients with SCD and the impact on both quality of life and physiological markers of health.
Eleven’s data and analytics create a longitudinal dataset of live biometrics and quality of life measures against metrics such as sleep, disease events, healthcare utilisation, and the impact of treatment pathways. Through this, Eleven builds a greater understanding of the disease in order to identify ways in which to improve outcomes for patients.
While much of their initial work has focused upon patients with SCD, it is an approach with translatability across the continuum of chronic and rare diseases – expansion into oncology and nephrology is already in the early stages of development. The biomarkers, records, and patient-reported outcomes Eleven uses are ones that have significant utility in a range of conditions, with monitoring quality of life forming an essential part of following the patient journey and treatment pathway.
The patient voice
‘We believe that the patient voice is a vital part of advancing patient care, better outcomes, and new medicines.’
To that end, a core part of Eleven focuses upon driving ongoing engagement with their patient community through a dedicated team who ensure all feedback is heard – be this about Eleven itself, their technology, patient health care and experiences, or the issues that affect them.
‘As we continue to learn more about patient experiences each day, this helps us to build a picture of what does and does not work well in terms of their care and treatments, in addition to helping to drive improvements to their interactions with Eleven.’
As Eleven Health applies their ecosystem to supporting life science partners in bringing their innovations one step closer to regulatory approval, a critical part of success is continued patient engagement and satisfaction. Already, their patient community has expressed significant benefits to their day-to-day life and overall wellbeing – helping to cement ongoing retention throughout the development of deep data insights and real-world evidence to drive improvements in their care and available therapeutic options. As a member of Eleven’s powerful patient community says:
‘I feel like I am contributing to the understanding of Sickle Cell Disease. Using my data to measure and potentially improve outcomes is really valuable for me and patients like me. Being able to potentially predict a crisis or VOC will really change things for Sickle Disease patients and I am excited to be part of this programme.’
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